Patients with the lysosomal storage disease mucopolysaccharidosis IIIA (MPSIIIA) lack the
lysosomal enzyme N-sulfoglucosamine sulfohydrolase (SGSH), one of the many enzymes …

Mucopolysaccharidosis type IIIA (MPS-IIIA) is a severe neurodegenerative lysosomal
storage disorder caused by a deficiency of N-sulfoglucosamine sulfohydrolase (SGSH) …

For most lysosomal storage diseases (LSDs) affecting the CNS, there is currently no cure.
The BBB, which limits the bioavailability of drugs administered systemically, and the short …

Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme
sulfamidase (SGSH), which is responsible for the degradation of heparan sulfate (HS). Build …

Abstract Mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome) is a lysosomal
storage disorder caused by the congenital deficiency of sulfamidase (SGSH) enzyme and …

Gene therapy is a promising therapeutic alternative for Lysosomal Storage Disorders (LSD),
as it is not necessary to correct the genetic defect in all cells of an organ to achieve …

Abstract Background Mucopolysaccharidosis type IIIA (MPS IIIA) is the most common of the
mucopolysaccharidoses. The disease is caused by a deficiency of the lysosomal enzyme …

Mucopolysaccharidosis type IIIA is a severe degenerative disease caused by an autosomal
recessive defect of a gene encoding a lysosomal heparan-N-sulfamidase, the N …

Many viral backbones have been used as gene transfer vectors. However, the efficacy of
therapy based on human-derived vectors may be limited by the high incidence of pre …

Mucopolysaccharidosis type II (MPS II) is a rare X-linked genetic disorder caused by
deficiency of the lysosomal enzyme iduronate-2-sulfatase (IDS), leading to impaired …